The Hill: A hearing brought to tears over Right to Try legislation by Joseph V. Gulfo

Last week, Sen. Ron Johnson (R-Wis.) introduced new Right to Try (RTT) legislation intended to prevent federal agencies from interfering with or blocking the implementation of RTT laws that have been passed in 28 states to date. I attended Johnson’s press conference announcing his RTT bill — the Trickett Wendler Right to Try Act of 2016 — and was given the opportunity to speak at the briefing on RTT and the Right to Try Act of 2015 (H.R. 3012), both of which took place in Washington on Tuesday, May 10 — National ALS Advocacy Day.

Johnson’s proposal mirrors H.R. 3012, which is sponsored in the House by Rep. Matt Salmon (R-Ariz.). The language of the Senate bill is quite straightforward:

[T]he Federal Government shall not take any action to prohibit or restrict (1) the production, manufacture, distribution, prescribing, or dispensing of an experimental drug, biological product, or device that (A) is intended to treat a patient who has been diagnosed with a terminal illness; and (B) is authorized by, and in accordance with, State law. …

[N]o liability shall lie against a producer, manufacturer, distributor, prescriber, dispenser, possessor, or user of an experimental drug, biological product, or device for the production, manufacture, distribution, prescribing, dispensing, possession, or use of an experimental drug, biological product, or device that is in compliance with [the statute]. …

[T]he outcome of any production, manufacture, distribution, prescribing, dispensing, possession, or use of an experimental drug, biological product, or device that was done in compliance with subsection (a) shall not be used by a Federal agency reviewing the experimental drug, biological product, or device to delay or otherwise adversely impact review or approval of such experimental drug, biological product, or device.

The last provision is critically important for drug companies, who develop and manufacture the experimental products. Their reticence to provide experimental drugs to patients who are not participating in formal clinical trials is driven mostly by the very real threat that the Food and Drug Administration (FDA) will suspend all studies of the compounds if a severe or unexpected adverse event were to happen in a compassionate use setting, as occurred with CytRx Corp. Additionally, companies fear that adverse events and other outcomes that occur in these uncontrolled settings could hamper interactions with the FDA and have a severe deleterious impact on the development course and timetable, as well as the approval and final labeling of experimental drugs.

Joining Johnson at the event announcing his proposed legislation were Tim Wendler and Matt Bellina. Wendler’s wife, Trickett, died of ALS (amyotrophic lateral sclerosis) in March of 2015. She was 39 years old — married for just 10 years and balancing a career and three young children — when she was suddenly unable to walk in the middle of a business trip. Two months later, she was diagnosed with ALS. As Trickett states in a moving video, “I was literally taking Zumba classes in March and in a wheelchair by July.” Trickett’s father also had ALS and succumbed to his disease. They also share something else in common: Both were treated with the same drug, Rilutek (riluzole), which was approved in 1995, and slows the progression of the disease, but is not curative. Tim stated that “in a terminal illness, opportunities for hope are few and far between … and having a bill like this introduced gives an opportunity for hope.” He is worried for his three children who are predisposed for developing ALS by virtue of having the inherited gene.

There are experimental drugs in clinical trials for ALS; however, the entry criteria are quite restrictive. Therefore, compassionate-use requests are needed for the vast majority of patients who cannot obtain access to the studies. State RTT and the national bills look to expand the FDA’s compassionate-use program. But the FDA maintains that RTT poses a significant threat to patient safety.

Bellina, a 32-year-old former Navy fighter pilot who was diagnosed with ALS in 2012 when he was 30 years old and is now confined to a wheelchair, doesn’t see the safety issue when patients are terminal. He said, “we fight on principles for life, liberty and the pursuit of happiness … our principles are not to provide an abundance of caution, or the illusion of safety … we’re about giving people a chance to do what they think is best for them.” He closed by saying, “In this great country of ours, I am allowed to take my own life, in some states, but I can’t try an experimental treatment that may save me.”

Frank Mongiello, who developed ALS in October 2015 and is now wheelchair-bound and has great difficulty articulating due to muscle weakness of his jaw and tongue, also spoke. He drew a parallel to Abraham Lincoln’s quote, “If I am killed, I can die but once; but to live in constant dread of it, is to die over and over again,” by stating “I have an 80 chance to be dead in two years … seeing these experimental drugs and not being able to take them is like dying over and over again.”

Mongiello and is wife have six children, the youngest of which are 10-year-old twins. As v said, “We don’t have the luxury of time … I have six children, a wife and great family and I am not ready to leave them.” When Mongiello finished, there was not a dry eye in the packed room. As compelling as anything Frank labored greatly to say were two words on a bumper-sticker affixed to the back of his wheelchair: “ALS Sucks.”

On Monday, May 16, the Reagan Udall Foundation for the FDA conducted a public meeting to discuss a navigator, a “coordinated resource for clear information to help patients and healthcare providers navigate the Expanded Access (EA) request process, also known as compassionate use. The focus of the EA Navigator would be (non-emergency) individual patient access to investigational drugs (“single patient INDs”).”

But a navigator is not what terminally ill patients need: With organizations like the Goldwater Institute, which has provided the model for state RTT laws, and patient advocacy and disease awareness groups, the knowledge of experimental drugs that may help patients is not at issue. In fact, the RTT movement itself is testimony to the fact that patients and their loved ones are quite savvy and capable of using the internet to find the experimental drugs. The real issues are the amount of time (100 hours) and paperwork (multiple submissions to the FDA and to institutional review boards), as well as the liability incurred by drug companies, doctors, hospitals and patients.

If the FDA truly supported compassionate use, it would fully endorse state RTT laws and the national acts introduced by Salmon and Johnson. Simultaneously, it would immediately enact its own guidance document (issued in February 2015) that reduces the amount of time required to complete a compassionate use request from 100 hours to 15 minutes. Interestingly, at yesterday’s navigator meeting, the FDA committed to implementing this guidance document “very, very soon.”

Actions speak louder than words, and the FDA’s actions with respect to RTT clearly demonstrate that the agency is loath to see the compassionate use program expanded in any meaningful manner. The House and Senate need to pass these national bills to give terminally ill patients a chance and a real measure of hope.

Gulfo is the executive director of the Rothman Institute of Innovation and Entrepreneurship at Fairleigh Dickinson University and author of “Innovation Breakdown: How the FDA and Wall Street Cripple Medical Advances” (Post Hill Press). He has more than 25 years of experience in the biopharmaceutical and medical-device industries and is the former CEO of Mela Sciences. Follow him on Twitter @josephgulfo.